Literature DB >> 27727217

Big win possible for Ionis/Biogen antisense drug in muscular atrophy.

Ken Garber.   

Abstract

Entities:  

Year:  2016        PMID： 27727217     DOI： 10.1038/nbt1016-1002

Source DB:  PubMed          Journal:  Nat Biotechnol        ISSN： 1087-0156            Impact factor:   54.908


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  8 in total

1.  Correction of disease-associated exon skipping by synthetic exon-specific activators.

Authors:  Luca Cartegni; Adrian R Krainer
Journal:  Nat Struct Biol       Date:  2003-02

2.  SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.

Authors:  James Palacino; Susanne E Swalley; Cheng Song; Atwood K Cheung; Lei Shu; Xiaolu Zhang; Mailin Van Hoosear; Youngah Shin; Donovan N Chin; Caroline Gubser Keller; Martin Beibel; Nicole A Renaud; Thomas M Smith; Michael Salcius; Xiaoying Shi; Marc Hild; Rebecca Servais; Monish Jain; Lin Deng; Caroline Bullock; Michael McLellan; Sven Schuierer; Leo Murphy; Marcel J J Blommers; Cecile Blaustein; Frada Berenshteyn; Arnaud Lacoste; Jason R Thomas; Guglielmo Roma; Gregory A Michaud; Brian S Tseng; Jeffery A Porter; Vic E Myer; John A Tallarico; Lawrence G Hamann; Daniel Curtis; Mark C Fishman; William F Dietrich; Natalie A Dales; Rajeev Sivasankaran
Journal:  Nat Chem Biol       Date:  2015-06-01       Impact factor: 15.040

3.  A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy.

Authors:  C L Lorson; E Hahnen; E J Androphy; B Wirth
Journal:  Proc Natl Acad Sci U S A       Date:  1999-05-25       Impact factor: 11.205

4.  Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.

Authors:  Nikolai A Naryshkin; Marla Weetall; Amal Dakka; Jana Narasimhan; Xin Zhao; Zhihua Feng; Karen K Y Ling; Gary M Karp; Hongyan Qi; Matthew G Woll; Guangming Chen; Nanjing Zhang; Vijayalakshmi Gabbeta; Priya Vazirani; Anuradha Bhattacharyya; Bansri Furia; Nicole Risher; Josephine Sheedy; Ronald Kong; Jiyuan Ma; Anthony Turpoff; Chang-Sun Lee; Xiaoyan Zhang; Young-Choon Moon; Panayiota Trifillis; Ellen M Welch; Joseph M Colacino; John Babiak; Neil G Almstead; Stuart W Peltz; Loren A Eng; Karen S Chen; Jesse L Mull; Maureen S Lynes; Lee L Rubin; Paulo Fontoura; Luca Santarelli; Daniel Haehnke; Kathleen D McCarthy; Roland Schmucki; Martin Ebeling; Manaswini Sivaramakrishnan; Chien-Ping Ko; Sergey V Paushkin; Hasane Ratni; Irene Gerlach; Anirvan Ghosh; Friedrich Metzger
Journal:  Science       Date:  2014-08-08       Impact factor: 47.728

5.  Identification and characterization of a spinal muscular atrophy-determining gene.

Authors:  S Lefebvre; L Bürglen; S Reboullet; O Clermont; P Burlet; L Viollet; B Benichou; C Cruaud; P Millasseau; M Zeviani
Journal:  Cell       Date:  1995-01-13       Impact factor: 41.582

6.  Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.

Authors:  Yimin Hua; Timothy A Vickers; Hazeem L Okunola; C Frank Bennett; Adrian R Krainer
Journal:  Am J Hum Genet       Date:  2008-03-27       Impact factor: 11.025

7.  Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.

Authors:  Yimin Hua; Kentaro Sahashi; Frank Rigo; Gene Hung; Guy Horev; C Frank Bennett; Adrian R Krainer
Journal:  Nature       Date:  2011-10-05       Impact factor: 49.962

8.  Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.

Authors:  Claudia A Chiriboga; Kathryn J Swoboda; Basil T Darras; Susan T Iannaccone; Jacqueline Montes; Darryl C De Vivo; Daniel A Norris; C Frank Bennett; Kathie M Bishop
Journal:  Neurology       Date:  2016-02-10       Impact factor: 9.910
  8 in total
  9 in total

Review 1.  The chemical evolution of oligonucleotide therapies of clinical utility.

Authors:  Anastasia Khvorova; Jonathan K Watts
Journal:  Nat Biotechnol       Date:  2017-02-27       Impact factor: 54.908

2.  Spinal muscular atrophy approval boosts antisense drugs.

Authors:  Elie Dolgin
Journal:  Nat Biotechnol       Date:  2017-02-08       Impact factor: 54.908

3.  Gene therapy rescues newborns with spinal muscular atrophy.

Authors:  Cormac Sheridan
Journal:  Nat Biotechnol       Date:  2018-08-06       Impact factor: 54.908

Review 4.  How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy.

Authors:  N N Singh; M D Howell; E J Androphy; R N Singh
Journal:  Gene Ther       Date:  2017-05-09       Impact factor: 5.250

Review 5.  Nusinersen: First Global Approval.

Authors:  Sheridan M Hoy
Journal:  Drugs       Date:  2017-03       Impact factor: 9.546

Review 6.  The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders.

Authors:  Philippine Aupy; Lucía Echevarría; Karima Relizani; Aurélie Goyenvalle
Journal:  Biomedicines       Date:  2017-12-22

Review 7.  Viral Vector-Mediated Antisense Therapy for Genetic Diseases.

Authors:  Marine Imbert; Gabriella Dias-Florencio; Aurélie Goyenvalle
Journal:  Genes (Basel)       Date:  2017-01-26       Impact factor: 4.096

Review 8.  Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders.

Authors:  Alireza Shahryari; Marie Saghaeian Jazi; Saeed Mohammadi; Hadi Razavi Nikoo; Zahra Nazari; Elaheh Sadat Hosseini; Ingo Burtscher; Seyed Javad Mowla; Heiko Lickert
Journal:  Front Genet       Date:  2019-09-25       Impact factor: 4.599

9.  Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation.

Authors:  Yifat S Oren; Michal Irony-Tur Sinai; Anita Golec; Ofra Barchad-Avitzur; Venkateshwar Mutyam; Yao Li; Jeong Hong; Efrat Ozeri-Galai; Aurélie Hatton; Chen Leibson; Liran Carmel; Joel Reiter; Eric J Sorscher; Steve D Wilton; Eitan Kerem; Steven M Rowe; Isabelle Sermet-Gaudelus; Batsheva Kerem
Journal:  J Cyst Fibros       Date:  2021-07-02       Impact factor: 5.482
  9 in total

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